uniQure Hemophilia B Deal With CSL Leaves M&A Fans Miffed
Dutch Group Gets $450m Upfront For Gene Therapy
Observers have been expecting uniQure to be sold off as a whole but the Netherlands-based firm has chosen to sell just one asset - EtranaDez - and focus on advancing its Huntington’s disease gene therapy.
You may also be interested in...
The gene therapy EtranaDez for hemophilia B placed on clinical hold after a patient was diagnosed with liver cancer, but UniQure points to that patient’s extensive history of liver disease.
The company plans to file with 52-week data for AMT-061 and is confident it won't face the same stringent requirements from the FDA that led to a complete response letter for BioMarin's Roctavian.
BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.